2017 Agenda

Dr. Pollastri’s current research has been broadly focused on addressing parasitic disease by repurposing known classes of investigational agents that have been successfully pursued for other indications. For example, entire families of kinase inhibitors discovered for cancer indications can often be redirected and optimized for potency against parasite cells. Dr. Pollastri and his team’s efforts to repurpose kinase-targeting chemotypes as new lead compounds for neglected tropical diseases will be presented. In particular, their model of distributed drug discovery, which involved pharma industry collaborators, will be highlighted.

Michael Pollastri, Ph.D., Associate Professor, Chair of Chemistry & Chemical Biology, Northeastern University

 

Asieris Pharmaceuticals is a biotech company focusing on transmuting knowledge from old drugs for the development of innovative drugs. APL-1202, an obsolete antibiotic approved for urinary tract infections, is currently in a pivotal clinical trial in China for non-muscle invasive bladder cancer (NMIBC). Using APL-1202 and its 2^nd-generation APL-1301 as examples, the presentation tries to elucidate Asieris’ approaches of how to capitalize the advantages while addressing the challenges from drug repositioning.

Kevin Pan, Ph.D., Co-Founder, Asieris Pharmaceuticals

 

In this presentation, Dr. Austin will discuss the following:

♦ Overview of NCATS New Therapeutic Uses Programs

♦ Challenges in need of solutions

♦ Crowdsourcing audience ideas

Bobbie Ann Austin, Ph.D., Program Officer, Drug Development Partnership Programs, National Center for Advancing Translational Sciences (NCATS), NIH, Office of the Director

 

Recursion's proprietary discovery platform brings together artificial intelligence with automated high throughput biology for therapeutic repurposing for rare genetic diseases. I will describe how we leverage these sophisticated screening methods to interrogate drugs against human disease models at scale.

Ron Alfa, MD, Ph.D., Vice President, Discovery and Product, Recursion Pharmaceuticals

 

A new R&D platform pioneered by Pharnext, PLEOTHERAPY™, allows the identification and development of synergistic combinations of repositioned drugs at low doses. This approach is universal as it is applicable to any diseases and any compound whether in clinical development or off patent. A proof-of concept of PLEOTHERAPY™ was obtained with the lead PLEODRUG™, PXT3003, in Charcot-Marie-Tooth disease type 1A (CMT1A) through positive Phase 2 results. An international pivotal Phase 3 clinical trial is currently ongoing in 30 centers across Europe, the U.S. and Canada. PXT864, Pharnext’s second lead PLEODRUG™, with a novel mechanism of action, completed a Phase 2a for Alzheimer’s disease with positive encouraging data. In this presentation, Mr. Paoli and Dr. Hajj will:

 

♦ Describe the PLEOTHERAPYTM R&D platform based on network pharmacology

♦ Illustrate the proof of concept of this platform through the presentation of both clinical lead projects: CMT1A and Alzheimer’s disease

♦ Introduce Pharnext’s business model and growth strategy for years to come

Xavier Paoli, CCO and VP of R&D Operations, PharNext SA

Rodolphe Hajj, Ph.D., Chief Pharmacology Officer, PharNext SA 

 

Vidula Sukhatme, Founder & CEO, Global Cures

 

The Story of Fenfluramine in Dravet Syndrome
In this presentation Dr. Legae will discuss his team’s involvement in the discovery of the SCN1A mutation in Dravet syndrome; how they found out that the few photosensitive patients responding to fenfluramine actually were children with Dravet; how they then designed their clinical study (paper Epilepsia 2012); how they are now working together with Zogenix to get fenfluramine on the market; and how they started translational research in a zebrafish model to confirm the efficacy. 
Liven Lagae, MD, Ph.D., Professor, Paediatric Neurology, Katholieke Universiteit Leuven, President, European Society for Paediatric Neurology

 

Cures Within Reach for Cancer aims to fund and facilitate the development of repurposed generic drugs for cancer. In this presentation, Dr. Kleiman will discuss plans for 1) a KnowledgeBase to systematically assess and prioritize repurposing opportunities, 2) Phase II/III clinical trials, and 3) advocacy for the treatments to be incorporated into standard of care so all cancer patients can access and benefit from them. 

Laura Kleiman, Ph.D., Executive Director, Cures Within Reach for Cancer 

 

Pairnomix will address the convergence of genetic sequencing, patient-specific mutation models, and comprehensive drug repurposing screening to identify new therapeutic options in rare disease. They will present a case study for Nav1.6 sodium channels (SCN8A) as well as additional ongoing work in other ion channels, and will discuss future directions for therapy development beyond genetic epilepsy.

Gregory Stewart, Ph.D., Chief Scientific Officer, Pairnomix

Computational methods are powerful tools for drug repositioning, and many such successful approaches are built around the complex network paradigm. Indeed, available drug datasets are very appropriate for describing structural relationships between pharmaceutical active substances such as chemical similarities or drug-target interactions, as well as behavioral connections such as drug-drug interactions. In this presentation, Dr. Bogdan will show how to extract previously unaccounted pharmacological properties using only drug-drug interaction networks.  By employing algorithmic clustering of drug-drug interaction networks, we recover recent and old repositionings but we can also indicate new potential drug repurposings. 

Paul Bogdan, Ph.D., Assistant Professor, Department of Electrical Engineering, University of Southern California